Compound record · Longevity & mitochondrial
Elamipretide
Also SS-31 · Forzinity · Bendavia · MTP-131 · D-Arg-Dmt-Lys-Phe-NH2
Mitochondria-targeting tetrapeptide (cardiolipin binder)
Mitochondrial peptide approved for one rare disease; negative trials elsewhere
Record reviewed 1 September 2026 · 6 references
- Class
- Mitochondria-targeting tetrapeptide (cardiolipin binder)
- Route
- Daily subcutaneous injection
- Approved use
- Barth syndrome ≥ 30 kg — US accelerated approval, Sept 2025
- Trial participants
- > 450 across phase 2/3 programmes; primary endpoints mostly not met
- Prescription
- Required in the US; not authorised in UK, EU, AU or CA
01 Overview
What Elamipretide is
Summary
Elamipretide is a synthetic tetrapeptide that concentrates in the inner mitochondrial membrane and binds cardiolipin, with the aim of stabilising the electron transport chain and improving energy production. In September 2025 the FDA granted it accelerated approval as Forzinity to improve muscle strength in people with Barth syndrome weighing at least 30 kg — a rare genetic mitochondrial disease — based on an intermediate endpoint, with a confirmatory trial required. Phase 3 trials in primary mitochondrial myopathy, dry age-related macular degeneration and heart failure did not meet their primary endpoints, and there are no studies in healthy adults.
Mechanism
Binds cardiolipin in the inner mitochondrial membrane, which is proposed to preserve cristae structure, improve coupling of the electron transport chain, increase ATP production and reduce the formation of reactive oxygen species. Whether these effects translate into clinical benefit has only been accepted by a regulator for Barth syndrome, and then on an intermediate endpoint.
02 Evidence by goal
What has been shown, for which goal
Grades follow one scale across the site. Limited overall means: small or short human studies, mixed results, or evidence mainly for a related use.
Longevity
InsufficientMitochondrial decline is a proposed driver of ageing, but elamipretide has never been studied for healthy ageing. Its approved indication is a rare genetic disease, and the largest trial in acquired mitochondrial myopathy (n = 218) was negative on its primary endpoints.
Athletic performance
InsufficientNo studies in athletes or healthy adults. In primary mitochondrial myopathy, 24 weeks of 40 mg daily did not improve six-minute walk distance versus placebo (MMPOWER-3).
Muscle & recovery
InsufficientThe approved indication is muscle strength in Barth syndrome, based on knee-extensor strength in 12 patients. This does not extend to muscle recovery or growth in people without mitochondrial disease.
General wellbeing
InsufficientFatigue scores did not improve versus placebo in the phase 3 mitochondrial myopathy trial; there are no data on energy or fatigue in people without a mitochondrial disorder.
03 Regulatory status
Where it is authorised, and for what
Status is recorded per jurisdiction from regulator sources and reviewed by hand. It is never inferred from another region's decision.
United Kingdom
Not authorisedNo MHRA marketing authorisation; available only in clinical trials or via unlicensed routes.
The MHRA agreed a paediatric investigation plan for elamipretide in Barth syndrome in January 2026 (applicant Atnahs Pharma UK), which is a development step, not a licence. No marketing authorisation had been granted at the time of this review.
04 Dosing research
What the evidence says about exposure
Published human studies and the doses, routes and durations they used — reported as research information, not a recommendation.
Research information — not a recommendation. These are the exposures used in published human studies, reported so you can see what has been tested. They are not dosing instructions and do not apply to any individual.
Study 01
TAZPOWER — elamipretide in Barth syndrome (the approval trial)
Reid Thompson W et al., Genet Med 2021;23:471–478
- Design
- Randomised, double-blind, placebo-controlled crossover (two 12-week periods, 4-week washout) followed by an open-label extension
- Population
- Genetically confirmed Barth syndrome, aged ≥ 12 years, weighing > 30 kg
- Participants
- n = 12
- Duration
- 12 weeks per crossover period; open-label extension to 168 weeks or more
- Route
- Subcutaneous injection
- Doses studied
- 40 mg once daily by subcutaneous injection
- Outcome at this exposure
- The crossover part did not meet its primary endpoints (six-minute walk distance and fatigue score). Improvements in walk distance, fatigue and knee-extensor strength were reported during the uncontrolled open-label extension from week 36 onwards, and the FDA accepted knee-extensor strength as an intermediate endpoint for accelerated approval.
- Adverse events observed
- Injection-site reactions in essentially all participants (erythema 100% vs 25% on placebo; pain 75% vs 42%; induration and pruritus 67% vs 17%; bruising and urticaria 25% vs 0%).
Study 02
MMPOWER-3 — elamipretide in primary mitochondrial myopathy
Karaa A et al., Neurology 2023;101:e238–e252 · Source
- Design
- Randomised, double-blind, placebo-controlled
- Population
- Adults with genetically confirmed primary mitochondrial myopathy (74% mtDNA, 26% nuclear DNA defects)
- Participants
- n = 218
- Duration
- 24 weeks
- Route
- Subcutaneous injection
- Doses studied
- 40 mg once daily by subcutaneous injection
- Outcome at this exposure
- Did not meet either primary endpoint: six-minute walk distance difference −3.2 m versus placebo (95% CI −18.7 to 12.3) and no difference in total fatigue score. A pre-specified subgroup with nuclear DNA defects showed improvement in walk distance, prompting further study.
- Adverse events observed
- Most adverse events mild to moderate; injection-site reactions were the most frequent. Treatment was described as well tolerated.
Study 03
ReCLAIM-2 — elamipretide in dry age-related macular degeneration
Ophthalmology 2025 (published online November 2024); NCT03891875 · Source
- Design
- Randomised (2:1), double-masked, placebo-controlled, multicentre
- Population
- Adults ≥ 55 years with dry AMD and non-central geographic atrophy
- Participants
- n = 176
- Duration
- 48 weeks
- Route
- Subcutaneous injection
- Doses studied
- 40 mg once daily by subcutaneous injection
- Outcome at this exposure
- Primary endpoints (low-luminance visual acuity and geographic atrophy area) were not met. Progression of ellipsoid-zone attenuation was reduced by about 43–47% and more patients gained ≥ 10 letters (14.6% vs 2.1%) — nominal, secondary findings.
- Adverse events observed
- Adverse events in 86% of elamipretide versus 71% of placebo participants, most commonly injection-site reactions (pruritus, pain, bruising, erythema).
Every trial used pharmaceutical-grade elamipretide at 40 mg subcutaneously once daily (or intravenous infusion in early cardiac studies) in people with a diagnosed mitochondrial, retinal or cardiac disease. No study has examined 'SS-31' in healthy adults, athletes or for ageing, and 'research chemical' vials are not the approved product.
05 Safety
Adverse effects and contraindications
Common effects seen in trials or reports, serious effects that warrant urgent review, and conditions under which use is not appropriate or needs assessment.
Common adverse effects
- Injection-site erythema (redness) — reported in all Barth syndrome trial participants
- Injection-site pain, induration, itching, bruising or hives
- Dizziness
- Headache
- Nausea
Serious adverse effects
- Hypersensitivity reactions (serious hypersensitivity is the only labelled contraindication)
- Benzyl alcohol toxicity in neonates ('gasping syndrome') — the approved formulation contains a preservative
- Accumulation in severe kidney impairment without dose adjustment
- Unknown long-term effects in people without mitochondrial disease — no data
Contraindications
- Kidney diseaseCaution
Exposure rises by up to 125% in severe renal impairment; the US label halves the dose (20 mg daily) at eGFR < 30 mL/min and gives no recommendation for people on dialysis.
Do not use = should not be used · Caution = needs assessment
06 Interactions
Medicine classes that need review
Grouped by how seriously the combination should be taken. Class labels match the medicines questionnaire in the assessment.
- Major
- Moderate
- Minor
Minor
Generally compatible; awareness is sufficient.
Anti-inflammatory painkiller
Ibuprofen, naproxen, diclofenac
No clinically significant pharmacokinetic interactions are identified in the US label. Because exposure depends on kidney function, medicines that can reduce renal function are a theoretical consideration only.
Diuretic
Furosemide, bendroflumethiazide
No interaction identified in the label; listed only because dehydration or reduced kidney function would be expected to raise elamipretide exposure.
07 Pregnancy & breastfeeding
Status in pregnancy
No human data. Animal studies at the exposures tested did not show adverse developmental effects, but the US label notes the absence of adequate data in pregnancy and breastfeeding. The product contains benzyl alcohol and must not be used in neonates.
08 Monitoring
What is usually monitored
Parameters that trials and product information track. A clinician decides what applies to an individual.
- 01Kidney function (eGFR) before starting and periodically, with dose adjustment if impaired
- 02Injection sites — rotate daily and watch for persistent reactions
- 03An objective functional outcome agreed in advance (for example a timed walk or strength test)
- 04Any signs of hypersensitivity after injection
- 05Fatigue and exercise tolerance recorded against a baseline
09 Combinations
What is known about combining it
Notes on pairing with other compounds in the directory: whether the combination has been studied in people, and where mechanisms overlap.
MOTS-c
No human studies
Overlap · Both are mitochondria-targeted peptides.
No human data on combining them; elamipretide's approved use is a rare mitochondrial disease and MOTS-c has no human efficacy data.
Epitalon
No human studies
Overlap · Both are marketed as 'cellular ageing' peptides.
No human data on the combination; neither has evidence for healthy ageing.
BPC-157
No human studies
No human data on this combination; BPC-157 itself lacks human efficacy data.
CJC-1295
No human studies
Marketed together in 'energy and recovery' stacks; no human data on the combination.
Discuss any proposed combination with a qualified healthcare professional.
10 Source considerations
Supply, quality and legitimacy
How the compound reaches people in practice, and what that means for product quality.
- 01In the US, Forzinity is a prescription-only medicine supplied for Barth syndrome; it is not available for wellness or performance use.
- 02'SS-31' vials sold online as research chemicals are not the approved product and lack the quality assurance, preservative system and concentration (80 mg/mL) of Forzinity.
- 03Outside the US there is no authorised product; anything offered is unlicensed and unverified.
11 Questions for your clinician
Take these to your appointment
Specific to this compound. The personal assessment adds questions drawn from your own history and medicines.
- 01Do I have a diagnosed mitochondrial disorder, or is my interest in 'mitochondrial support' for general fatigue?
- 02How should the negative phase 3 results in mitochondrial myopathy, AMD and heart failure inform expectations for my goal?
- 03How is my kidney function, and would the labelled dose adjustment apply to me?
- 04What would daily injections for months realistically involve in terms of injection-site reactions?
- 05Which fatigue causes with authorised treatments — thyroid, iron, sleep, mood — should be excluded first?
The personal assessment tailors this list to your responses.
12 References
Sources behind this record
Regulator documents and peer-reviewed publications used to derive every grade and statement above.
- 01FDA news release — Accelerated approval of Forzinity for Barth syndrome (19 September 2025)
- 02Forzinity (elamipretide) US Prescribing Information (2025)
- 03Reid Thompson W et al. TAZPOWER — elamipretide in Barth syndrome. Genet Med 2021;23:471–478
- 04Karaa A et al. MMPOWER-3 — elamipretide in primary mitochondrial myopathy. Neurology 2023
- 05ReCLAIM-2 — elamipretide in dry AMD. Ophthalmology 2025
- 06EMA orphan designation EU/3/21/2430 — elamipretide for Barth syndrome
The Peptide Checkup provides educational information and a structured summary of published research and regulatory status. It is not medical advice, does not diagnose or treat any condition, and does not replace a consultation with a qualified healthcare professional.
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